There are about 25,000 patients with MG in the USA.
According to the ( I agree with Annie,very limited) literature, about 1/3 will get significantly better, about 1/3 will stay the same (or get somewhat better) and about 1/3 will get worse. (in the past those would die, but now they are kept alive with the much better supportive and symptomatic care that we have got. Not
because there is better treatment for the illness itself).
So 1/3-2/3 of the patients are overall content with the current management of MG and can lead a normal or near-normal life. They will never experience any significant breathing problems and will ( I could argue that some of them are over-treated, but that is a different issue or maybe not?).
1/3 have a severe debilitating and some even have a potentially life-endangering illness (this means that their life can be saved if they are given adequate respiratory support on time, which is fortunately
what usually happens, but unfortunately
So, in the USA alone there are about 8000 MG patients whose life has been significantly disrupted by this illness. ( you can do the math to see how many there are world-wide).
This is a very small number in a population of 300 million, but this is not a small absolute
number. Each of those people has family, friends and co-workers who are effected by their illness.
Depending on the approach of their treating physicians and those patient's own preferences- Some of them will receive aggressive (and experimental) treatments, (such as would normally be given to patients with cancer, including stem cell transplants etc.), some of them will be told to seek psychiatric treatment, some of them will find innovative ways to live with their illness and some of them will possibly die. (yes, you can die from MG. Even the most optimistic reviews say there is about a 5% mortality rate in respiratory crisis and about 20% of MG patients will have a crisis during the course of their illness. so do the math. It is not zero).
Those statistics do not include "un-diagnosed" MG patients.
If we assume that about 5% of MG patients will have normal test results, and if we assume that about 90% of them will not be diagnosed as MG (I am basing this on a rough estimate that 1/10 neurologists are ready to diagnose MG without "objective" tests), then there should be about another 1000 MG patients whose illness is not properly managed (and not even properly diagnosed).
None of the 25,000 MG patients receive treatment which was adequately tested and was shown to be of benefit which outweighs the risk in similar patients.
I have given before the example of immune thrombocytopenia. (an autoimmune disease in my field of practice).
In the past (20 years ago) all the patients were treated with immunosupressive treatment.
We now know that some patients (with a mild disease) will never require treatment and the outcome of those who are treated is worse than that of those who are not, because both will do well, but those who are treated will have needless complications from the treatment itself.
Some patients require aggressive treatment from the very early stages of their illness.
Some patients will have a complete remission with a short course of steroids, but others will not do well when the dose is tapered. So, we now have ways to know who should be given steroids and who will require high doses for long periods and therefore will overall not benefit from this treatment.
Some patients will benefit from a surgical approach, whereas others will not and we choose the patients accordingly.
Some patients (a minority) will have a refractory disease and not respond to any of our commonly used treatments.
That is why we keep on doing clinical trials to find better management approaches for those patients.
of this is being done in MG.
Why? there can be many explanations-it is much easier to check the number of platelets than assess a complex neurological disease like MG. Hematologists don't (normally) attribute their patient's lack of response to negative thoughts or not putting enough efforts. A low platelet count doesn't effect a person's functional abilities, so patients with ITP don't bother their physicians as much and mostly do lead a completely normal life in between exacerbations/ hospitalizations. Physicians taking care of patients with ITP have a much better understanding of the immune system because they also take care of patients with lymphoid malignancies.
But, the reasons don't matter.
There is no reasonable excuse for such a major difference between the management of one autoimmune disease to the management of another autoimmune disease. There is no reasonable explanation why over the last 20 years there have been major changes in the way ITP is managed and no such changes in MG. There is no reason why hematologists are still concerned about the small minority of refractory patients (and therefore conduct clinical trials to find better ways to manage their illness) and neurologists (even excellent ones) do not show similar concerns about their patients. There is no reason why hematologists are concerned about over-treating their patients and neurologists have no problem giving high doses of medications with significant side-effect profiles for extended periods.
I think it is time to stop asking "why?"
Annie is right. It doesn't really matter.
It's time to ask "what"
can be done to change this.
I am sorry if I am annoying some people who prefer to think that MG is an ultra-rare disease, that there is already enough knowledge about it, that there is no place for further research and why should we care about those few people who have to struggle every day to do the most basic things and sometimes can't even breath.
This is a movie done by a group of patients with CFS.
I believe it had a significant impact. It did on me. It made me realize that CFS is not what I was told it is.
Patients can change the way their illness is viewed by the medical community and the media, patients can influence how much research will be done on their illness.
We can't blame neurologists for being content with the way things are, when we as patients who receive such treatment are content with it.
The M.D. Anderson cancer center is going to put 3 billion dollars in finding better ways to treat different cancers in the next decade.
2 of them are in my field of practice, and there are already numerous clinical trials trying to do that world-wide.
What is the planned budget for such research on MG?